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A couple of weeks ago I started participating in my first drug trial. It is for a drug called fingolimod that is already approved for treating multiple sclerosis. It was approved by the FDA around the time I was diagnosed and after talking to people at a number of different clinics, it seemed like the most promising trial available near me. It helps regulate certain immune cells related to damage to motor neurons. There is anecdotal evidence that it helps with ALS, with some patients even seeing their progressions halt. It has also been shown beneficial to rats with a form of ALS, so it must work on me right?

One of the possible side effects is that it can cause heart arrhythmias. So before I started on the drug, I had to go through some health screening. Of course this was more complicated than it should have been because my EKG showed some abnormalities, so I had to cardiologists make sure I didn’t have any heart issues. Those tests turned out fine and I was able to start the trial. So far I haven’t noticed any of the scary potential side effects. I guess that’s a good thing, but maybe it means I am on the placebo.

Because this is the first ALS trial for fingolimod, it is just a one month safety trial with only 30 participants. Their main focus is tracking whether or not it causes nasty side effects like fainting or heart problems. Even if it turns out that it works on ALS patients in slowing or halting the progression of the disease, I am not sure if one month will be long enough for me to see a big difference. They will track my lung function and use a questionnaire to try and see if it changes my progression, but luckily I would guess both of those would be fairly constant for me over a one-month time frame with or without the drug. My lung function hasn’t changed over the past 9 months and I have noticed a change in function, but not day to day or week to week, but over the course of a few months.

If fingolimod is shown to be safe, they will follow up with a year long trial, with more subjects, that should have a chance of demonstrating efficacy. However, unless they change the exclusion criteria I wouldn’t be able to participate. For this one, your symptoms must have started within the past two years. I’m not 100% sure when mine started, but I think two Christmases ago is a good estimate. Cara gave me a pair of non-skinny jeans that year and I had a lot of trouble getting my hands into the pockets. Looking further back than that, I probably was becoming a worse soccer player the year before, however it is hard to know how much to attribute to ALS versus just being fat and out of shape.

But this raises a huge issue related to trials. The majority of people with ALS don’t qualify for current trials. Most restrict participation to people whose symptoms started in the last two or three years and people who aren’t too sick. Well, some people may experience symptoms for around a year before seeing a doctor (I waited at least that long). Then for many people it takes up to a year to get an accurate diagnosis (I was lucky and was diagnosed in a couple of months but know plenty of people who were repeatedly misdiagnosed over months or years). So most people with ALS have an extremely short window in which they can participate in trials.

After that, there aren’t really any other approved options. The one current ALS drug extends life expectancies for a few months. A few months better than nothing, but it is still a tough pill to swallow. Your health, your life, is just dependent on the randomness of your progression. You are left to depend on your faith and your vitamins to survive, even if there are potentially beneficial treatments going through the trial process.

The nice thing about fingolimod though, is that it is already FDA approved for MS. That means a doctor could prescribe it “off label” to an ALS patient. You can sit down with your doctor and after considering the risks, and the uncertainty of the drug’s effects, you can decide if you want to try it. For most investigational treatments this isn’t an option. Even if they have gone through safety trials, and have helped some patients, they can’t be prescribed until they are approved. It doesn’t matter if your doctor thinks it will work for you, or that without it, your prognosis is grim. You must wait for FDA approval. And this takes years. Too many ALS patients don’t have that kind of time. Many people live two to five years, so they don’t have the luxury of waiting for final approval. This is the issue that the ALS ETF (http://alsetf.org/) is trying to address.

The FDA allows investigational drugs that have evidence of safety and at least suggested evidence of efficacy to be made available to patients with life-threatening illnesses in situations where there are no other effective treatments. However, it must to be done through authorized expanded access programs (EAPs). No one has done this yet for ALS. It was done frequently in the 1990s for HIV treatments and has also been done for a number of cancer drugs. So here is something that could truly impact patients today and change the way things are done, and it doesn’t take convincing politicians to change laws. It just takes the resources and effort to make it possible. We are in the process of to raising funds to sponsor an EAP for an ALS treatment. We believe there are a number of investigational drugs that would qualify and would provide hope to patients who otherwise are left with few other options. There is obviously a huge need to continue to do basic research and to continue with well-controlled trials to find effective treatments but that does not mean we should be denying near-term treatments to current patients.

With fingolimod, the potential pathway was discovered in 2009. In July 2011, they had results from preclinical testing using rats. In February 2013, they got FDA approval for the trial and announced the four sites where they would conduct it. And then after repeatedly telling me they were one IRB form away from approval, it was another 9 months before Augusta enrolled me as their first patient. If all goes well, they would likely get marketing approval by 2015 or 2016 at the earliest. That’s the life time of a whole generation of ALS patients from start to finish. Expediting near-term treatments is a big deal. The FDA recognizes that finding a treatment for ALS is different than finding a new substitute for Viagra, and patients facing life threatening diseases are willing to accept different risks, so they allow for EAPs. Now someone needs to do it. ALS ETF is trying to fill that gap. It is a volunteer organization staffed by a team of people affected by ALS. The director’s background is in finance, but he has spent the past five years or so learning the ins and outs of the ALS community and becoming an expert on EAPs. He recently lost his father to ALS, but continues to believe that current patients do not need to be sacrificed while we wait for full marketing approval for the next ALS drug. Even if the treatments in the pipeline make it that far, we are still talking years before they are widely available. Too many patients don’t have that kind of time.

I apologize if this post is a little more “in the weeds” about dealing with ALS. Maybe I can’t make the case as well as others can. Our website now has an excellent explainer video (http://alsetf.org/) This just seems to be a hole in the current landscape that needs to be filled. Here is something that could immediately change the availability of options to patients and does not require convincing Congress to change laws to make it possible. It may be a no-brainer, buts a a no-brainer that someone has to act on to solve.

Don’t get me wrong, I still am extremely hopeful that effective treatments will be found. Some may currently be in the pipeline, while others will come from more basic research that is currently underway. I am still extremely thankful that my progression seems to be going slowly. I still love everything else about my life. I am still touched by the generosity of our friends. I may now have more opinions about ALS, but I am no less hopeful and am still blown away by people working to change the course of the disease, improving the lives of people living with the disease, and people living incredible lives in the face of the disease.

Over Thanksgiving, we were in Arlington visiting my parents and then Cara flew to Europe for the week for work. We felt that we were getting behind on Christmas prep, so I wanted to get a tree before she got back. Pulling a nine and a half foot tree off the roof of my car and standing up in our living room really isn’t in my wheelhouse right now so I had to ask a friend help me out. So instead of him going home to help with his toddler twins at home one night, he put up my Christmas tree. Little things mean a lot, and make a huge difference. His wife somehow finds time between chasing the kids and being a partner at a big law firm in town to make us casseroles. One less thing for us to worry about. It makes a difference.

A few weeks ago we went to a fundraiser for the SC Chapter of the ALS Association. It is an organization going to great lengths to support families dealing with ALS. I hadn’t quite realized it, but the idea of having this event in Columbia sprung from an email a good friend of mine wrote to SCALSA saying he would love to help put on an event. I met him through soccer and a local pub. When we first moved to Columbia, I played pick up soccer regularly. One week some guys invited me to play on their indoor team. We lost the first game, but afterwards some guys from the other team made the mistake of telling me that if I followed them back to the pub, the first beer would be on the house. Needless to say, I soon joined the pub’s team, and for the next couple years spent my Thursday nights playing indoor and then hanging out too late for a school night with this motley group of guys. Now the pub is a place where I can go watch a match and know I’ll bump into a good friend.

Early in the fall, I heard from SCALSA they were planning an event up here and I joined their planning committee. I was touched when I got to the first meeting and a bunch of my buddies from the pub were there. I didn’t even know they were familiar with the association. After a few months of meetings and planning by these guys and a big crew of additional volunteers (and new friends), the event went off without a hitch. A good turnout, plenty of food, a successful silent auction, and most importantly good times had by all. A big crew from Cara’s office showed up, and a hefty proportion of the O30/O40 Columbia soccer community. Even a bunch of guys who have plenty of reasons to avoid me like the plague! This means something. In a roomful of people like that no one affected by ALS could feel like they are in it alone.

After 9 months, I know more about ALS. I know there are things that should change. I know there is work to be done. And I know there are plenty of reasons for hope.

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3 thoughts on “On Trial and the role of the ALS Emergency Treatment Fund”

Hi There, I am so pleased to hear this news. I’m sending prayers this med works, they amend the exclusion criteria, and you can enter the year long trial. I am interested in learning more about the ALS ETF organization also. The info you are sharing has reenforced the sense of urgency for drugs to get to market without a doubt. I too wish this was quicker. Your family and Christmas tree are beautiful.

Thank you for sharing your story and making a difference in so so many things and ways.

Stephen, when we were in our late teens and your dad worried about what you’d do once you traded engineering for economics, I never doubted you would have a great life w a great wife and some great kids. I did, however, worry about your hair. Needlessly, it now seems as you’ve still got it. Well done. Xoxo Griff